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HD InsightsFeatured Story: Reflections on Twenty Years of Trials

Reflections on Twenty Years of Trials

By Victoria Segro, ANP-C

Vicki Segro is a nurse practitioner and a rater and sub-investigator at the Rocky Mountain Movement Disorders Center in Englewood, Colorado.

I have had the pleasure of participating in the care of patients with Huntington’s disease for the last 20 years, and now working with multiple generations of these families.

The approach in how we care for patients participating in clinical trials is certainly different compared to routine clinical care, but the quality of the care should be the same. In our work, good care of a person with HD participating in a clinical trial appreciates both the science of the study and the human being willing to volunteer for the study.

The team members must all understand the course of clinical illness in HD to provide excellent care, of course. This includes the motor symptoms of HD, cognitive symptoms, as well as the emotional symptoms of the illness. This becomes more challenging as the science behind the studies becomes more complex over time.

Understanding the basics of the science is critical. “God is in the details,” they say. When I was learning to participate in clinical trials as a nurse practitioner, I was assisted by a seasoned research coordinator who impressed on me how important it was to pay attention to the details, and helped me learn how to do that in the context of a study. This saved me and others from rework, embarrassment, trial delays, and most importantly, potential harm to participants.

Organization skills are a must, particularly scheduling visits within time windows. We have some studies that we pre-schedule over six months ahead to keep within those windows. Our office schedules a mix of routine clinic follow-up patients, new clinic patients, and study participants on every day of the week, so scheduling far in advance is preferable.

Caring at the Center of It All

The best study coordinators I have worked with have a deep commitment to the care of the entire family that is affected by HD. They are in “for the long haul” with a long-term commitment to HD. I think this leads to a greater willingness for patients to commit to participation in studies. We must always remind ourselves that patients with HD and their families bring a lot of hope and commitment to this – they are providing us with the ability to perform the research through volunteerism.

Personal relationships often come with the territory. Some research studies allow us to spend more time with patients and their caregivers because of more frequent office visits through the course of the study. As a result, you learn more about their personal interests and, in some cases gain a deeper understanding of how HD affects their lives and relationships.

We may then connect people to resources, if needed, through the local Huntington’s Disease Society of America or other community groups. I am amazed by the extent families will go to to provide care for their loved one –whether a parent, sibling, or a child. More frequent contact with patients allows me to appreciate their commitment to research and finding a cure for this disease.

I once had a gene-positive, asymptomatic patient who was participating in an observational study tell me about the anxiety that came with annual visits. Each visit, she worried that there would be a formal diagnosis of HD. It made me realize that even observational studies put a lot of stress on participants. I can now have open conversations which validate anxiety and fear over coming in for what may be somewhat routine visit.

I remember a young lady who came to our site to administer the first open label (no placebo) dose of Tominersen in the GEN-EXTEND study. She was uncomfortable because this was a single visit at our site and she had never met any of our staff. The visit and the procedure went smoothly, for which I was thankful. At the end of her visit, the excitement and hope that she and her family expressed was overwhelming. I was grateful to have participated in her care that day. This kind of experience affirms the whole reason I love to participate in research.

A Partner to the Provider

Our research coordinators spend a great deal of time with patients and caregivers and then report important interval history events to the provider prior to their being seen by the provider. Tracking medication changes and health events that occur between visits is tedious in some cases, particularly when the patient may have seen other providers in the interim.

Providers greatly appreciate our organizing the study tasks during the visit so they may complete their portion efficiently. It isn’t the most exciting aspect of the job, but everyone wins when you double-check that the paperwork is properly completed, with everything signed and dated correctly.

My advice is to do your job as though there will be no second set of eyes on it – then get a second set of eyes on it! I cannot count the times that I filled in the wrong date on paperwork, particularly in the new year, and the study coordinator helped me to fix it right away.

Bonding Around Tasks and the HD Mission

Communication with the larger study team is vital. We have monthly research meetings with all of the coordinators and providers in the office. We discuss study recruitment as well as larger working issues related to each individual study. In addition, study coordinators have a separate monthly meeting to discuss specific details and coordinator specific challenges.

After we have enrolled a few patients into a new study protocol, the coordinators and providers become more comfortable with the “flow” of procedures. We can expect some organized chaos for the first patient participating in their first study visit, but somehow, we manage to get through it all in one piece. I have learned to expect this over time and we will counsel participants that this may happen. To minimize the snafus, our seasoned HD study coordinators mentor new study coordinators and coordinators transitioning care from patients with other diagnoses to HD.

In this process, a cohesive team begins to develop. Further strengthening it, teams also participate in local community events and fundraisers. It is a pleasure getting to participate in hoop-a-thons and walks and other local fundraising activities. These activities help build a strong team of caregivers and creates an excellent work environment.

Meeting a Ballooning Complexity Challenge

The biggest challenge of research in HD is trying to keep up with the science behind the research, which is moving at a faster pace over time. This includes observational studies, studies of new medications for the treatment of symptoms of HD, and new treatments meant to slow the progression of this disease. We are studying medications that may be administered via many routes, including pill forms, intravenous infusions, intrathecal infusions (through a lumbar puncture process), and even through direct injection into the brain.

Every year, the science is advancing to provide better treatments of symptoms of HD with fewer side effects. Ultimately, the goal of treatment is to slow or even stop the progression of HD. I look forward to the future when we see the day that we find a cure and will be proud to have contributed to the cause.

About HD Insights

Our mission is to promote, disseminate, and facilitate research on Huntington’s disease. To fulfill this mission, we are guided by an outstanding editorial board that includes representatives from three continents, academia, industry, and the HD community.